EMA rejects FDA-approved Parkinson's drug, signs off on Moderna vaccine use in adolescents ahead of FDA
EMA said it considered that the results of the clinical studies used to support the application “were inconsistent and did not satisfactorily show that Nouryant was effective at reducing the ‘off’ time. Only four out of the eight studies showed a reduction in ‘off’ time, and the effect did not increase with an increased dose of Nouryant.”
So-called “off” periods occur when a Parkinson’s patient has difficulty moving about and occur when the effect of the last dose of another common Parkinson’s drug, known as levodopa, wears off.
“The Agency also noted that no effect was seen in the two studies that included patients from EU populations, including the most recent study which involved patients who were receiving the maximum and optimal treatment for their Parkinson’s disease,” EMA said.
The FDA in announcing its approval noted that four, 12-week placebo-controlled clinical studies that included a total of 1,143 participants showed Nourianz offered a statistically significant decrease from baseline in daily “off” time compared to those on placebo.
Meanwhile, EMA’s Committee for Medicinal Products for Human Use adopted a positive opinion recommending marketing authorization for Moderna’s Covid-19 vaccine, now known as Spikevax, to include adolescents 12 years of age and older. The use of the Spikevax vaccine in children from 12 to 17 years of age will be the same as in people aged 18 and above, EMA said.
Moderna filed an application with the FDA for that same younger age group on June 10 but has yet to hear from the agency. States like Rhode Island have already signed off on allowing 12 to 17-year-olds to use the vaccine.
CHMP also recommended for approval Sanofi and Genzyme’s new Pompe disease drug, known in Europe as Nexviadyme (avalglucosidase alfa). Pompe disease is a rare, inherited disorder caused by the buildup of glycogen in the body’s cells.
EMA said the drug can help improve the respiratory function of Pompe disease patients, and the most common side effects include hypersensitivity (including anaphylaxis) and infusion-associated reactions. In the US, avalglucosidase alfa saw its PDUFA date pushed back by three months, from May 18 to Aug. 18, according to a Sanofi investor presentation in April.
Though the program obtained priority review from FDA back in November, analysts expressed skepticism in July 2020 after it failed to prove superior to the companies’ other drug Lumizyme in improving respiratory function in a Phase III trial.
CHMP on Friday also announced that Roche pulled its Tecentriq application in triple-negative breast cancer after it noted that the EMA said the results from a late-stage trial do not favor the drug’s benefit-risk calculation in this indication. In the US, however, ODAC in April voted to keep alive the accelerated approval for Tecentriq plus Abraxane (nab-paclitaxel) in mTNBC while additional confirmatory trials are ongoing.
And finally, CHMP also concluded its investigation into bluebird bio’s Zynteglo, finding no evidence that it causes a blood cancer known as acute myeloid leukemia.
Earlier this month, the drugmaker lifted a voluntary EU marketing hold on Zynteglo for beta thalassemia, after a patient death in one of the drugmaker’s other products, LentiGlobin, halted clinical studies. Bluebird determined that death was highly unlikely to have been caused by the delivery virus used in both Zynteglo and LentiGlobin.
Bluebird also earned an approval Wednesday in Europe for its gene therapy Lenti-D for children with early cerebral adrenoleukodystrophy and an ABCD1 genetic mutation without a matched sibling donor.